Once certain that he would die young, a young man, born with the deadly disease, now dreams of growing old. “I’m going to be a grandfather someday. I’m going to have a really long life,” says Bill Elder, a 25-year-old Stanford University graduate who is now applying for medical school.
That’s because of a blue pill and a new trend in drug development called venture philanthropy.
Elder has cystic fibrosis (CF). It’s known as an “orphan disease” because so few people have it — only about 30,000 in the U.S. and about 70,000 worldwide — so there is little incentive for drug companies to seek a cure.
When Piper Beatty was born 30 years ago and diagnosed with CF at 6 weeks, health workers gave her parents a heartbreaking message: “Take her home and love her as long as you have her.” At that time, the best the family could hope for was that she would live to be a teenager. Beatty, now 30 and a law school graduate, has high hopes for improved therapies for CF patients and others born with rare genetic diseases.
Developing a single new drug can cost a billion dollars, so pharmaceutical companies want to create blockbusters for common diseases like Alzheimer’s or cancer to maximize the return on their investment.
“From a patient perspective, venture philanthropy is a remarkable strategy,” Beatty said. “It’s the best of business and the best of philanthropy coming together. It allows us to be very active in the drug development process. It allows people with a stake in the disease to get involved to partner with the drug companies. You take an active role at the forefront of the fight.”
Other charities including the Michael J. Fox Foundation, which is fighting for a cure for Parkinson’s, are following the CF model.